NCT05788536

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

A PHASE 1/2, OPEN-LABEL, MULTICENTER TRIAL WITH A SINGLE ASCENDING DOSE COHORT WITH UNILATERAL INTRACOCHLEAR INJECTION FOLLOWED BY A BILATERAL INJECTION EXPANSION COHORT TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF DB-OTO IN CHILDREN AND INFANTS WITH BIALLELIC hOTOF MUTATIONS

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

Summary

Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat children who have hearing loss due to changes in the otoferlin gene. The purpose of this study is to: * Learn about the safety of DB-OTO * Determine how well DB-OTO is tolerated (does not cause ongoing discomfort) * Evaluate the efficacy of DB-OTO (how well DB-OTO works)

Detailed description

Former Sponsor Decibel Therapeutics

Conditions

• Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Interventions

Type	Name	Description
GENETIC	DB-OTO	DB-OTO will be administered as a single intracochlear injection into one ear (Part A). * LD Cohort (lower dose) * HD Cohort (high dose) - not implemented
GENETIC	DB-OTO	DB-OTO will be administered as a single intracochlear injection into both ears (Part B). For bilateral injections (Part B), patients will receive DB-OTO in 1 surgical session.

Timeline

• Start date: 2023-06-27
• Primary completion: 2031-04-19
• Completion: 2031-04-19
• First posted: 2023-03-29
• Last updated: 2026-02-23

Locations

15 sites across 4 countries: United States, Germany, Spain, United Kingdom

Regulatory

• FDA-regulated drug study